Episode 1: FDA Advisory Board’s Concerns About New Duchenne Drug, Brain Fog in Immune Thrombocytopenia; August is SMA Awareness Month
The CheckRare Brief delivers a concise summary of the week's most important developments in rare diseases. Each episode covers FDA approvals, clinical trial updates, conference highlights, scientific publications, and industry news that matter to healthcare professionals, researchers, advocates, and the rare disease community.Madaline Spencer, Podcast Producer; James Radke, PhD; Education Director; Peter Ciszewski, Founder and CEO, CheckRare References for this episode’s topics: FDA Advisory Report on Capricor Therapeutics’ Orphan Drug to Treat Cardiomyopathy in Duchenne Muscular DystrophyFDA…
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